Item 1A. Risk Factors
Risks Related to Our Financial Position and Need for Additional
Capital
We have a history of operating losses,
may incur additional operating losses in the future and may never achieve sustained profitability.
Our prospects must be considered
in light of the uncertainties, risks, expenses and difficulties frequently encountered by companies in the early stages of operation.
We incurred net losses of approximately $17.9 million and $46.3 million for the years ended December 31, 2024 and 2023, respectively.
As of December 31, 2024, we had an accumulated deficit of approximately $339.6 million. We expect to incur substantial additional operating
expenses over the next several years as our research, development, pre-clinical testing, clinical trial and commercialization activities
increase as we commercialize DefenCath and develop our other product lines. As a result, we may experience negative cash flow at times
as we fund our operating expenses and capital expenditures. Our ability to generate revenue and maintain profitability will depend on,
among other things, the following: successfully continued marketing and selling DefenCath in the U.S.; obtaining and/or maintaining reimbursement
for DefenCath in appropriate settings of care; obtaining necessary regulatory approvals for our other products from the FDA and, if sought,
international regulatory agencies; establishing additional manufacturing, sales, and marketing arrangements, either alone or with third
parties; and raising sufficient funds to finance our activities if we are unable to generate sufficient revenue from the commercialization
of DefenCath in the U.S. We might not succeed at any of these undertakings. If we are unsuccessful at some or all of these undertakings,
our business, prospects, and results of operations may be materially adversely affected.
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We may need to finance our future cash
needs through public or private equity offerings, debt financings or corporate collaboration and licensing arrangements. Any additional
funds that we obtain may not be on terms favorable to us or our stockholders, may dilute our stockholders, and may require us to relinquish
valuable rights.
To date, our commercial operations
have not generated sufficient revenues to enable profitability on an annual basis. We estimate that we have sufficient cash to fund (i)
operations for at least twelve months from the date of issuance of this Annual Report on Form 10-K and (ii) the ongoing commercial marketing,
sale and promotion of DefenCath. These estimates are based upon the base case assumptions for market penetration, average selling price,
research and development (“R&D”) expense and commercial infrastructure cost.
We may need additional financing to the extent we are unable to generate
sufficient revenue from the commercialization of DefenCath in the U.S. We can provide no assurances that any financing or strategic relationships
will be available to us on acceptable terms, or at all. We expect to continue to use significant cash to fund our operations as we commercialize
DefenCath in the U.S, pursue development of our other product lines and other business development activities, and potentially incur additional
legal costs to defend our intellectual property.
To raise needed capital,
we may sell additional equity or debt securities, obtain a bank credit facility, or enter into a corporate collaboration or licensing
arrangement. The sale of additional debt securities, if convertible, could result in dilution to our stockholders. The incurrence of
indebtedness would result in fixed obligations and could also result in covenants that would restrict our operations. Raising additional
funds through collaboration or licensing arrangements with third parties may require us to relinquish valuable rights to our technologies,
future revenue streams, research programs or product lines, or to grant licenses on terms that may not be favorable to us or our stockholders.
To the extent we raise additional
capital by issuing equity securities, our stockholders may experience substantial dilution. We may, as we have in the past, sell common
stock, convertible securities or other equity securities in one or more transactions at prices and in a manner we determine from time
to time. If we sell common stock, convertible securities or other equity securities in more than one transaction, investors may be further
diluted by subsequent sales. New investors could gain rights superior to existing stockholders.
Risks Related to the Commercialization
of DefenCath
We are highly dependent on the continued
successful commercialization of our only approved product, DefenCath.
Our ability to generate operating
revenue is dependent upon our continued successful commercialization of DefenCath in the U.S. DefenCath was approved by FDA on November
15, 2023, and is indicated to reduce the incidence of CRBSIs in adult patients with kidney failure receiving chronic hemodialysis through
a CVC. This drug is indicated for use in a limited and specific population of patients. We launched DefenCath commercially in April 2024
in the inpatient setting and in July 2024 in the outpatient hemodialysis setting. The safety and effectiveness of DefenCath have not
been established for use in populations other than adult patients with kidney failure receiving chronic hemodialysis through a CVC.
We have not commercialized
any product lines other than DefenCath. Continued successful commercialization of DefenCath is subject to many risks, including but not
limited to:
● ongoing maintenance of regulatory approvals;
● emergence of superior or equivalent products;
● failure to achieve significant market acceptance adoption.
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There is no guarantee that
our continued commercialization efforts will be successful, or that we will be able to successfully launch and commercialize any other
product lines that receive regulatory approval.
The continued successful commercialization
of DefenCath will depend on maintaining coverage and reimbursement for use of DefenCath from third-party payors.
Sales of pharmaceutical products largely depend on the reimbursement
of patients’ medical expenses by government health care programs, such as Medicare, Medicaid and/or private health insurers. Further,
significant uncertainty exists as to the reimbursement status of newly approved health care products. We currently sell DefenCath directly
to hospitals and outpatient dialysis center operators, but also may expand its usage into oncology and total parenteral nutrition patients
requiring catheters if those indications can be secured from the FDA. For any new indication of use, all new potential customers are healthcare
providers who depend upon reimbursement by government and commercial insurance payors for dialysis and other treatments. Depending on
the treatment setting of any new indication for use, we believe that DefenCath would be eligible for coverage under various reimbursement
programs, such as the IPPS, including certain temporary payment adjustments (e.g., NTAP); however, payment under these payment systems
could later be modified or decreased by future regulations. Further, CMS, which administers Medicare, and works with states to administer
Medicaid, has adopted and will continue to adopt and/or amend rules governing reimbursement for specific treatments. We anticipate that
insurers may increasingly demand that manufacturers demonstrate the cost effectiveness of their products as part of the reimbursement
review and approval process. Healthcare reform proposals and medical cost containment proposals designed to target rising healthcare costs
could be introduced in the U.S. Any measures affecting the reimbursement programs of governmental and private insurance payors, including
any uncertainty in the medical community regarding their nature and effect on reimbursement programs, could have an adverse effect on
purchasing decisions regarding DefenCath, as well as limit the price we may charge for DefenCath. The failure to obtain or maintain reimbursement
coverage for DefenCath or any other products could materially harm our operations.
In anticipation that payers
may increasingly demand that we demonstrate the cost effectiveness of DefenCath as part of the reimbursement review and approval process,
we have submitted posters and abstracts to support our health economic analysis and continue to commission and develop health economic
evaluations to support this review. We are pursuing opportunities to work with healthcare systems to demonstrate the clinical and economic
effectiveness of DefenCath; however, our studies might not be sufficient to support coverage or reimbursement at levels that allow providers
to use DefenCath.
We have significant customer concentration,
with a limited number of customers accounting for a large portion of our revenues.
We derive a large portion
of our revenues from a few major customers. Sales to one customer accounted for 86% of our total revenue for the year ended December
31, 2024, and we had two customers that accounted for 87% and 12% of our accounts receivable, respectively, for the year ended December
31, 2024. These customers have no purchase commitments and may cancel, change or delay purchases with little or no notice or penalty.
As a result of these customer concentrations, our revenue could fluctuate materially and could be materially and disproportionately impacted
by purchasing decisions of these customers or any other significant customer. These customers may decide to purchase less DefenCath from
us than management anticipates, may alter purchasing patterns at any time with limited notice, or may decide not to continue to purchase
DefenCath at all, any of which could cause our revenue to decline materially and materially harm our financial condition and results
of operations. If we are unable to diversify and grow our customer base, we will continue to be susceptible to risks associated with
customer concentration.
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Risks Related to the Development and Commercialization
of our Other Products
Successful development and commercialization
of new product lines is uncertain.
Our development and commercialization
of our product, and future product lines, is subject to the risks of failure and delay inherent in the development of new pharmaceutical
products, including but not limited to the following:
● inability to produce positive data in pre-clinical and clinical trials;
● challenges with securing the supply chain for raw materials;
● failure to receive or maintain regulatory approvals;
● emergence of superior or equivalent products;
● failure to achieve market acceptance.
Because of these risks, our
development efforts may not result in any future commercially viable products. If a significant portion of these development efforts
are not successfully completed, required regulatory approvals are not obtained or any approved products are not commercialized successfully,
our business, financial condition, and results of operations could be materially harmed.
Final approval by regulatory authorities
of our product lines for commercial use may be delayed, limited or denied, any of which could adversely affect our ability to generate
operating revenues.
The clinical development,
manufacturing, labeling, packaging, storage, recordkeeping, export, marketing, promotion and distribution, and other possible activities
relating to our product lines are subject to extensive regulation by the FDA and other regulatory agencies. Failure to comply with applicable
regulatory requirements may, either before or after product approval, subject us to administrative or judicially imposed sanctions that
may negatively impact the approval of one or more of our product lines or otherwise negatively impact our business. Compliance with such
regulations may consume substantial financial and management resources and expose us and our collaborators to the potential for other
adverse circumstances which could delay or prevent us from generating revenue from the commercialization of these drugs and cause us
to incur significant additional costs.
We are not permitted to market
a product line in the United States until the particular product line is approved for marketing by the FDA. Specific pre-clinical data,
chemistry, manufacturing and controls data, a proposed clinical trial protocol and other information must be submitted to the FDA as
part of an investigational new drug (“IND”) application, and clinical trials may commence only after the IND application
becomes effective. To market a new drug in the United States, we must submit to the FDA and obtain FDA approval of an NDA. An NDA must
be supported by extensive clinical and pre-clinical data, as well as extensive information regarding chemistry, manufacturing and controls,
to demonstrate the safety and effectiveness of the product line, and the FDA will also assess whether the manufacturing processes and
facilities are suitable to support the application. Approval of an NDA may be delayed due to delays in FDA’s review of the manufacturing
facility, which may require an onsite inspection.
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Obtaining approval of an
NDA can be a lengthy, expensive and uncertain process. Review time can be impacted by the quality of the information included in the
application, FDA’s internal resources such as the availability of reviewers, or requests from the FDA for additional information.
Regulatory approval of an NDA is not guaranteed. The number and types of pre-clinical studies and clinical trials that will be required
for FDA approval varies depending on the product line, the disease or condition that the product line is designed to target and the regulations
applicable to any particular product line. Despite the time and expense exerted in pre-clinical and clinical studies, failure can occur
at any stage, and we could encounter problems that delay our product line development or that cause us to abandon clinical trials or
to repeat or perform additional pre-clinical studies and clinical trials. The FDA can delay, limit or deny approval of a product line
for many reasons, and product line development programs may be delayed or may not be successful for many reasons including but not limited
to, the following:
● the FDA may change its approval policies or adopt new regulations.
Our pre-clinical and clinical
data, other information and procedures relating to a product line may not be sufficient to support approval by the FDA or any other U.S.
or foreign regulatory authority, or regulatory interpretation of these data and procedures may be unfavorable. Failure to conduct required
post-approval studies, or confirm a clinical benefit, will allow the FDA to withdraw the drug from the market on an expedited basis.
Our business and reputation may be harmed by any failure or significant delay in receiving regulatory approval for the sale of any drugs
resulting from our product lines. As a result, we cannot predict when or whether regulatory approval will be obtained for any drug we
develop.
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Additionally, other factors
may serve to delay, limit or prevent the final approval by regulatory authorities of our product lines for commercial use, including,
but not limited to:
The successful development
of any product lines is uncertain and, accordingly, we may never commercialize any of these product lines or generate significant revenue.
Risks Related to Healthcare Regulatory
and Legal Compliance Matters
Our approved product, DefenCath, is, and
our other product lines (if approved) will be, subject to extensive post-approval regulation.
Once a product is approved,
numerous post-approval requirements apply in the United States. These include, among other things, requirements related to pharmacovigilance
and adverse event and other reporting, supply chain security requirements, suspect and illegitimate product investigations and notifications,
limitations on product advertising and promotion and on the distribution of product samples, required post-marketing studies, and ongoing
adherence to cGMPs, as well as the need to submit appropriate new or supplemental applications and obtain FDA approval for certain changes
to the approved product, product labeling, or manufacturing process. Establishing and maintaining systems and procedures for compliance
with these requirements, and for training and monitoring personnel relative to their compliance, is expensive, time consuming, and an
ongoing effort. Depending on the circumstances, failure to meet post-approval requirements can result in criminal prosecution, fines,
injunctions, recall or seizure of products, total or partial suspension of production, denial or withdrawal of pre-marketing product
approvals, or refusal to allow us to enter into supply contracts, including government contracts. In addition, even if we comply with
FDA, foreign and other requirements, new information regarding the safety or effectiveness of a product could lead the FDA or a foreign
regulatory body to modify or withdraw product approval. Failure to complete a PREA post-marketing study can result in a PREA non-compliance
letter, which is publicly posted on FDA’s website, and could result in the product being considered misbranded and subject to additional
enforcement.
Current healthcare laws and regulations
in the U.S. and future legislative or regulatory reforms to the U.S. healthcare system may affect our ability to commercialize DefenCath
and future marketed products profitably.
Federal and state governments
in the U.S. are considering legislative and regulatory proposals to change the U.S. healthcare system in ways that could affect our ability
to commercialize DefenCath and future marketed products profitably. Similarly, among payors and other third-parties, there is significant
interest in promoting such changes through legislation and regulation (in additional to through restrictions introduced via contracting
and other methods). The life sciences industry and specifically the market for the sale, insurance coverage and distribution of pharmaceuticals
has been a particular focus of these efforts and would likely be significantly affected by any major legislative or regulatory initiatives.
In addition, there have been, and may in the future be, initiatives at both the federal and state level that could significantly modify
the terms and scope of government-provided health insurance coverage, ranging from changes to some or all of the provisions of existing
law, to establishing a single-payer, national health insurance system, to more limited “buy-in” options to existing public
health insurance programs, any of which could have a significant impact on the healthcare industry. It is possible that additional legislative,
executive and judicial activities in the future could have a material adverse impact on our business, financial condition and results
of operations.
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Healthcare policy changes, including reimbursement
policies for drugs and medical devices, may have an adverse effect on our business, financial condition and results of operations.
Our future revenues, profitability
and access to capital will be affected by the continuing efforts of governmental and private third-party payors to manage, contain or
reduce the costs of health care through various means, such as capping prices, limiting price increases, reducing reimbursement, and
requiring rebates. Market acceptance and sales of DefenCath or any other product lines that we develop, will depend on reimbursement
policies and may be affected by health care reform measures in the U.S. and abroad. Government authorities and other third-party payors,
such as private health insurers, decide which drugs they will pay for and establish reimbursement levels. While DefenCath has been approved
for reimbursement in certain settings, we cannot be sure that reimbursement will be available for DefenCath by other payers. That uncertainty
applies for any other product lines that we develop. Also, we cannot be sure that the amount of reimbursement that is available will
not reduce the demand for, or the price of, our products. If reimbursement is not available by certain payors or is available only at
limited levels, we may not be able to continue to successfully commercialize DefenCath or any other product lines that we develop.
In the U.S. there has been,
and we expect there will continue to be, a number of legislative and regulatory changes to the health care system that could affect our
ability to profit from our approved products. The U.S. government and other governments have shown significant interest in pursuing healthcare
reform. Any such government-adopted reform measures may adversely affect the pricing of healthcare products and services in the U.S.
or internationally and the amount of reimbursement available from governmental agencies or other third-party payors.
In recent years, the U.S.
Congress has sought to repeal and has significantly amended the Affordable Care Act. We expect that there will continue to be proposals
by legislators at both the federal and state levels, regulators and third-party payors to keep healthcare costs down while expanding
individual healthcare benefits. Certain of these changes could impose limitations on the prices we will be able to charge for any products
that are approved or the amounts of reimbursement available for these products from governmental agencies or other third-party payors
or may increase the tax requirements for life sciences companies such as ours. Any such changes could have an adverse effect on our business,
financial condition and results of operations.
There has been heightened
governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which have resulted in several recent
congressional inquiries and proposed and enacted bills by Congress and the states designed to, among other things, bring more transparency
to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement
methodologies for products. In addition, the U.S. government, state legislatures, and foreign governments have shown significant interest
in implementing cost containment programs, including price-controls, restrictions on reimbursement and requirements for substitution
of generic products for branded prescription drugs to limit the growth of government paid health care costs. For example, the U.S. government
has passed legislation requiring pharmaceutical manufacturers to provide rebates and discounts to certain entities and governmental payors
to participate in federal healthcare programs. The U.S. government enacted the Inflation Reduction Act of 2022 (Inflation Reduction Act
or IRA). The IRA brought sweeping changes to Medicare coverage and reimbursement for prescription drugs that could negatively impact
us and other pharmaceutical manufacturers. Of note, beginning January 1, 2025, the eliminates the Medicare Part D coverage gap, and reduces
a beneficiary’s out-of-pocket maximum to $2,000. The existing coverage gap discount program for pharmaceutical manufacturers will
be replaced by a new manufacturer discount program effective in 2025. Under the new program, manufacturers will provide a 10 percent
discount off the negotiated price for applicable drugs (branded drugs and biologics manufactured by companies that have Part D discount
agreements) after the deductible is satisfied through the catastrophic phase of the benefit. In the catastrophic phase, manufacturers
will provide a 20 percent discount off negotiated price.
Any reduction in reimbursement
rates under Medicare, Medicaid, or private insurers could negatively affect the pricing of our products. If we are not able to charge
a sufficient amount for our products, then our margins and our profitability will be adversely affected.
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Risks relating to data privacy could create
additional liabilities for us.
We are subject to data privacy
and protection laws and regulations that apply to the collection, transmission, storage and use of personally-identifying information.
Failure to comply with applicable privacy and data security laws and regulations could result in enforcement actions against us, including
possible fines, imprisonment of company officials and public censure, claims for damages by affected individuals, damage to our reputation
and loss of goodwill, any of which could have a material adverse effect on our business, financial condition, results of operations or
prospects.
The legislative and regulatory
landscape for privacy and data protection continues to evolve in jurisdictions worldwide. There are numerous U.S. federal and state laws
and regulations related to the privacy, data protection and security of personal information. At the federal level, regulations promulgated
pursuant to HIPAA establish privacy and security standards for “covered entities” (group health plans and most healthcare
providers) that limit the use and disclosure of individually identifiable health information those entities and their service providers
receive or create (“protected health information”). Although we generally are not subject to the HIPAA privacy or security
regulations, we do business with various entities (including clinical trial investigators) that are subject those regulations, and we
have to expend resources to understand their obligations, adjust contractual terms in light of those obligations, or otherwise modify
our business practices. Any amendments to HIPAA or other legislation amending or broadening the scope of HIPAA might require us to make
substantial expenditures and would likely create additional liability risks.
The Federal Trade Commission
(“FTC”) has used its authority under Section 5 of the FTC Act, which prohibits unfair and deceptive practices affecting consumers,
to bring numerous cases against companies for failing to protect the privacy or security of personal information in a manner that is
reasonable and fully consistent with stated privacy policies, notices, or other representations. The FTC has considered codifying its
requirements in regulations, but has not done so; as a result, the optimal means to mitigate the risk of such an action are uncertain.
In addition, many U.S. states
in which we operate have laws that protect the privacy and security of personal information. Certain state laws may be more stringent
or broader in scope, or offer greater individual rights, with respect to personal information than federal, international or other state
laws, and such laws may differ from each other, which complicates compliance efforts. For example, the California Confidentiality of
Medical Information Act (the “CMIA”) imposes stringent data privacy and security requirements and obligations with respect
to the personal health information of California residents. The CMIA authorizes administrative fines and civil penalties of up to $25,000
for willful violations and up to $250,000 if the violation is for purposes of financial gain, as well as criminal fines. Other states,
including California, Colorado, Connecticut, Delaware, Indiana, Iowa, Montana, New Hampshire, New Jersey, Oregon, Tennessee, Texas, Utah,
and Virginia, have recently adopted broadly applicable privacy laws, though these laws typically exempt personal health information or
entities that handle personal health information pursuant to laws like HIPAA. Both Nevada and Washington State have enacted laws specifically
to protect the privacy of health information. Violations of the Washington State law can result in civil penalties of up to $7,500 per
violation, up to $25,000 in treble damages at the sole discretion of the court, and injunctive relief. Consumers also may bring their
own actions to recover (i) actual damages, (ii) treble damages; and (iii) attorney’s fees. Violations of the Nevada law can result
in up to $10,000 civil penalties per violation and injunctive relief.
New legislation may be enacted
in other states. The effects on our business of this growing body of privacy and data protection laws are potentially significant, and
may require us to modify our data processing practices and policies and to incur substantial costs and expenses in an effort to comply.
If we or our third-party
service providers are unable to properly protect the privacy and security of personal information, or other confidential data we process
in our business, we could be found to have breached our contracts. Further, if we fail to comply with applicable privacy laws, we could
face civil and criminal penalties. Enforcement activity by regulatory authorities in relation to privacy and cybersecurity matters can
result in financial liability and reputational harm, and responses to such enforcement activity can consume significant internal resources.
The threat of class action lawsuits based on data security breaches or alleged unfair practices further increases the risk to our business.
We cannot be sure how these privacy laws and regulations will be interpreted, enforced or applied to our operations. In addition to the
risks associated with enforcement activities and potential contractual liabilities, our ongoing efforts to comply with evolving laws
and regulations at the federal and state level may be costly and require ongoing modifications to our policies, procedures and systems.
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Clinical trials required for our product
lines, including, but not limited to, new uses or formulations of DefenCath and the required DefenCath PREA study, may be expensive and
time-consuming, and their outcome is uncertain.
In order to obtain FDA approval
to market a new drug or device product, we must demonstrate proof of safety and effectiveness in humans. To meet FDA requirements, we
are obligated to conduct “adequate and well-controlled” clinical trials. Conducting clinical trials is a lengthy, time-consuming,
and expensive process. The length of time may vary substantially according to the type, complexity, novelty, and intended use of the
product line, and often can be several years or more per trial. Delays associated with the development plans for our product lines may
cause us to incur additional operating expenses. The commencement and rate of completion of clinical trials may be delayed by many factors,
including, for example:
● slower than expected rates of patient recruitment;
● failure to recruit a sufficient number of patients;
● modification of clinical trial protocols;
● changes in regulatory requirements for clinical trials;
● lack of effectiveness during clinical trials;
● emergence of unforeseen safety issues;
Further, the results
from early pre-clinical and clinical trials are not necessarily predictive of results to be obtained in later clinical trials. Accordingly,
even if we obtain positive results from early pre-clinical or clinical trials, we may not achieve the same success in later clinical
trials. Moreover, comparisons of results across different studies should be viewed with caution as such comparisons are limited by a
number of factors, including differences in study designs and populations. Such comparisons also will not provide a sufficient basis
for any comparative claims following product approval. Clinical results are frequently susceptible to varying interpretations that may
delay, limit or prevent regulatory approvals or commercialization. Negative or inconclusive results or adverse medical events during
a clinical trial could cause a clinical trial to be delayed, repeated or terminated, or a clinical program to be abandoned.
Our clinical trials may be
conducted in patients with serious or life-threatening diseases for whom conventional treatments have been unsuccessful or for whom no
conventional treatment exists, and in some cases, our product is expected to be used in combination with approved therapies that themselves
have significant adverse event profiles. During the course of treatment, these patients could suffer adverse medical events or die for
reasons that may or may not be related to our products. We cannot ensure that safety issues will not arise with respect to our products
in clinical development.
Clinical trials may not demonstrate
statistically significant safety and effectiveness to obtain the requisite regulatory approvals for product lines. The failure of clinical
trials to demonstrate safety and effectiveness for the desired indications could harm the development of our product lines. Such a failure
could cause us to abandon a product line and could delay development of other product lines. Any delay in, or termination of, our clinical
trials would delay the filing of any NDA or any Premarket Approval Application, or PMA, or De Novo application, with the FDA and, ultimately,
our ability to commercialize our product lines and generate product revenues. Any change in, or termination of, our clinical trials could
materially harm our business, financial condition, and results of operations.
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Changes in funding for the FDA and other
government agencies or future government shutdowns or disruptions could cause delays in the submission and regulatory review of marketing
applications, including supplements, which could negatively impact our business or prospects.
The ability of the FDA to
review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire
and retain key personnel and accept submission, applications, and the payment of user fees, and statutory, regulatory, and policy changes.
In addition, government funding of other government agencies that fund research and development activities is subject to the political
process, which is inherently fluid and unpredictable. The impact of the new administration, changes in federal policy priorities as well
as global events, including terrorism, natural disasters and pandemics, or other health emergencies, may also cause disruptions in the
normal functioning of the FDA or other government agencies.
Risks Related to Our Business and Industry
Healthcare institutions, physicians and
patients may not accept and use our products.
Even though we have received
FDA approval for DefenCath, healthcare institutions, physicians and patients may not accept and use our products. Acceptance and use
of our products will depend upon a number of factors including the following:
● prevalence of the disease to be treated or prevented;
● prevalence and severity of any side effects;
● cost-effectiveness of our product relative to current standard of care;
● timing of market introduction of our drugs and competitive drugs;
Because we expect sales of
DefenCath to generate substantially all of our product revenues for the foreseeable future, the failure of DefenCath to find market acceptance
would harm our business and would require us to seek additional financing.
Competition and technological change may
make DefenCath, as well as our other product lines or indications, less attractive or obsolete.
We compete with established
pharmaceutical and medical device companies that are pursuing other forms of prevention or treatment for the same or similar indications
we are pursuing, and that have greater financial and other resources. Other companies may succeed in developing products earlier than
we do, may develop products that are more effective than our product lines. Research and development by others may render our technology
or product lines obsolete or noncompetitive, or result in processes, treatments or cures superior to any therapy we develop. We face
competition from companies that develop competing technology internally, or acquire competing technology through acquisitions of other
companies, or from universities and other research institutions. As these competitors develop their technologies, they may develop competitive
positions that may prevent, make futile, or limit our product commercialization efforts, which would result in a decrease in the revenue
we would be able to derive from the sale of DefenCath or our other product lines if any of such other product lines receive marketing
approval.
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If we lose key management or scientific
personnel, cannot recruit qualified employees, directors, officers, or other personnel or experience increases in compensation costs,
our business may materially suffer.
We are highly dependent on
the principal members of our management and scientific staff, specifically, Joseph Todisco, our Chief Executive Officer, Dr. Matthew
David, our Executive Vice President and Chief Financial Officer, Beth Zelnick Kaufman, our Executive Vice President, Chief Legal and
Compliance Officer and Corporate Secretary, Elizabeth Hurlburt, our Executive Vice President and Chief Clinical Strategy & Operations
Officer and Erin Mistry, our Executive Vice President and Chief Commercial Officer. Our future success will depend in part on our ability
to identify, hire, and retain current and additional personnel. We experience intense competition for qualified personnel and may be
unable to attract and retain the personnel necessary for the development of our business. Because of this competition, our compensation
costs may increase significantly. In addition, we have only limited ability to prevent former employees from competing with us.
We may not successfully manage our growth.
Our success will depend upon
the expansion of our operations to continue to commercialize DefenCath and the effective management of any growth, which could place
a significant strain on our management and our administrative, operational and financial resources. To manage this growth, we may need
to expand our facilities, augment our operational, financial and management systems and hire and train additional qualified personnel.
Additionally, if market demand exceeds our third-party manufacturer’s ability to produce DefenCath, we may not be able to fulfill
our customers orders in a timely manner or at all, which may have an adverse impact on our results of operations and reputation. If we
are unable to manage our growth effectively, our business may be materially harmed.
If we are unable to effectively recruit,
train, retain and equip our sales force, our ability to continue successfully commercialize DefenCath will be harmed.
None of the newly hired members of our sales force has promoted DefenCath
before, and we are required to, and will continue to be required to, expend significant time and effort to train the sales force to be
credible, persuasive, and compliant with applicable laws in marketing DefenCath for its approved indication. We must train the sales force
to ensure that a consistent and appropriate message about DefenCath is being delivered to our customers. If we are unable to successfully
train the sales force and provide them with appropriate materials, including medical and sales literature to help them educate and inform
customers about the benefits and risks of DefenCath our efforts to continue to successfully commercialize DefenCath may be challenged.
We face the risk of product liability claims
and the amount of insurance coverage we hold now or in the future may not be adequate to cover all liabilities we might incur.
Our business exposes us to
the risk of product liability claims that are inherent in the development of drugs. If the use of one or more of our or our collaborators’
drugs or devices harms people, we may be subject to costly and damaging product liability claims brought against us by clinical trial
participants, consumers, health care providers, pharmaceutical companies or others selling our products.
We currently carry product
liability insurance. We cannot predict all of the possible harms or side effects that may result and, therefore, the amount of insurance
coverage we hold may not be adequate to cover all liabilities we might incur. Our insurance covers bodily injury and property damage
arising from our clinical trials, subject to industry-standard terms, conditions and exclusions. Our coverage also includes the sale
of commercial products.
If we are unable to obtain
insurance at an acceptable cost or otherwise protect against potential product liability claims, we may be exposed to significant liabilities,
which may materially and adversely affect our business and financial position. If we are sued for any injury allegedly caused by our
or our collaborators’ products and do not have sufficient insurance coverage, our liability could exceed our total assets and our
ability to pay the liability. A successful product liability claim or series of claims brought against us would decrease our cash and
could cause the value of our capital stock to decrease.
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We may be exposed to liability claims associated
with the use of hazardous materials and chemicals.
Our research, development
and manufacturing activities and/or those of our third-party contractors may involve the controlled use of hazardous materials and chemicals.
Although we believe that our safety procedures for using, storing, handling and disposing of these materials comply with federal, state
and local, as well as foreign, laws and regulations, we cannot completely eliminate the risk of accidental injury or contamination from
these materials. In the event of such an accident, we and the third-party could be held liable for any resulting damages and any liability
could materially adversely affect our business, financial condition and results of operations. In addition, the federal, state and local,
as well as foreign, laws and regulations governing the use, manufacture, storage, handling and disposal of hazardous or radioactive materials
and waste products may require us to incur substantial compliance costs that could materially adversely affect our business, financial
condition and results of operations.
Risks Related to Our Intellectual Property
If we and our licensors do not obtain protection
for and successfully defend our respective intellectual property rights, competitors may be able to take advantage of our research and
development efforts to develop competing products.
Our commercial success will
depend in part on obtaining further patent protection for our products, product lines and other technologies and successfully defending
any patents that we currently have or will obtain against third-party challenges. The patents which we currently believe are most material
to our business are as follows:
We may seek further patent
protection for our compounds and methods of treating diseases. However, the patent process is subject to numerous risks and uncertainties,
and there can be no assurance that we will be successful in protecting our products by obtaining and defending patents. These risks and
uncertainties include the following:
In addition, the USPTO and
patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related
inventions be limited or narrowed substantially to cover only the specific innovations exemplified in the patent application, thereby
limiting the scope of protection against competitive challenges. Thus, even if we or our licensors are able to obtain patents, the patents
may be substantially narrower than anticipated. Additionally, the breadth of claims allowed in biotechnology and pharmaceutical
patents or their enforceability cannot be predicted. We cannot be sure that, should any patents issue, we will be provided with adequate
protection against potentially competitive products. Furthermore, we cannot be sure that should patents issue, they will be of commercial
value to us, or that private parties, including competitors, will not successfully challenge our patents or circumvent our patent position
in the U.S. or abroad.
The above-mentioned patents
are exclusively licensed to or owned by us. To support our patent strategy, we have engaged in a review of patentability and certain
freedom to operate issues, including performing certain searches. However, patentability and certain freedom to operate issues are inherently
complex, and we cannot provide assurances that a relevant patent office and/or relevant court will agree with our conclusions regarding
patentability issues or with our conclusions regarding freedom to operate issues, which can involve subtle issues of claim interpretation
and/or claim liability. Furthermore, we may not be aware of all patents, published applications or published literature that may affect
our business either by blocking our ability to commercialize our product lines, preventing the patentability of our product lines to
us or our licensors, or covering the same or similar technologies that may invalidate our patents, limit the scope of our future patent
claims or adversely affect our ability to market our product lines. Additionally, it is also possible that prior art of which we
are aware, but which we do not believe affects the validity or enforceability of a claim, may, nonetheless, ultimately be found by a
court of law or an administration panel to affect the validity or enforceability of a claim. If a third-party were to prevail on a legal
assertion of invalidity and/or unenforceability, we would lose at least part, and perhaps all, of the patent protection on our product
lines. Such loss of patent protection could have a material adverse impact on our business. Additionally, since patent applications
in the United States are maintained in secrecy until published or issued and as publication of discoveries in the scientific or patent
literature often lag behind the actual discoveries, we cannot be certain that we were the first to make the inventions covered by the
pending patent applications or issued patents referred to above or that we were the first to file patent applications for such inventions.
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In addition to patents, we
also rely on trade secrets and proprietary know-how. Although we take measures to protect this information by entering into confidentiality
and inventions agreements with our employees, and some but not all of our scientific advisors, consultants, and collaborators, we cannot
provide any assurances that these agreements will not be breached, that we will be able to protect ourselves from the harmful effects
of disclosure or dispute ownership if they are breached, or that our trade secrets will not otherwise become known or be independently
discovered by competitors. We may also be unsuccessful in executing such an agreement with each party who in fact develops intellectual
property that we regard as our own, which may result in claims by or against us related to the ownership of such intellectual property.
If any of these events occurs, or we otherwise lose protection for our trade secrets or proprietary know-how, the value of our intellectual
property may be greatly reduced. Even if we are successful in prosecuting or defending against such claims, litigation could result
in substantial costs and be a distraction to our senior management and scientific personnel.
Intellectual property disputes could require
us to spend time and money to address such disputes and could limit our intellectual property rights.
The biotechnology and pharmaceutical
industries have been characterized by extensive litigation regarding patents and other intellectual property rights, and companies have
employed intellectual property litigation to gain a competitive advantage. We may initiate or become subject to infringement claims or
litigation arising out of patents and pending applications of our competitors, or we may become subject to proceedings initiated by our
competitors or other third parties or the PTO or applicable foreign bodies to reexamine the patentability of our licensed or owned patents.
In addition, litigation may be necessary to enforce our issued patents, to protect our trade secrets and know-how, or to determine the
enforceability, scope, and validity of the proprietary rights of others. If we are required to defend patent infringement actions brought
by third parties, or if we sue to protect our own patent rights, we may be required to pay substantial litigation costs and managerial
attention may be diverted from business operations even if the outcome is not adverse to us. In addition, any legal action that seeks
damages or an injunction to stop us from carrying on our commercial activities relating to the affected technologies could subject us
to monetary liability and require us or any third-party licensors to obtain a license to continue to use the affected technologies. We
cannot predict whether we would prevail in any of these types of actions or that any required license would be made available on commercially
acceptable terms or at all. Furthermore, to the extent that we or our consultants or research collaborators use intellectual property
owned by others in work performed for us, disputes may also arise as to the rights in such intellectual property or in resulting know-how
and inventions. An adverse claim could subject us to significant liabilities to such other parties and/or require disputed rights to
be licensed from such other parties. See Note 7, Commitments and Contingencies, of this Annual Report on Form 10-K for additional
detail on the Company’s legal proceedings.
Risks Related to Dependence on Third Parties
We depend on third-party suppliers and
contract manufacturers for the supply and manufacture of DefenCath and our product lines, as well as our APIs, which subjects us to potential
cost increases and manufacturing delays that are not within our control.
We do not manufacture DefenCath
or any of its raw materials or components ourselves, and we rely on third parties for our drug supplies both for clinical trials and
for commercial quantities. All of our manufacturing processes currently are, and we expect them to continue to be, outsourced to third
parties, some of which are single-source suppliers. We have made the strategic decision not to manufacture APIs for DefenCath or our
other product lines, as these can be more economically supplied by third parties with particular expertise in this area. We have engaged
contract facilities that are registered with the FDA, have a track record of large-scale API manufacture, and have already invested in
capital and equipment.
We currently have one FDA approved source for each of our two key APIs
for DefenCath, taurolidine and heparin sodium, respectively. With regards to taurolidine, we have a DMF filed with the FDA. There is a
master commercial supply agreement between a third-party manufacturer and the Company in place from August 2018. In addition, we are working
with our existing manufacture to source sufficient quantities of taurolidine API to cover at least 24 months of potential future demand.
With respect to heparin sodium API, we have identified an alternate third-party supplier and may qualify such supplier under the DefenCath
NDA over the next twelve months.
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We received FDA approval of DefenCath with finished dosage production
from our European based CMO Rovi Pharma Industrial Services. We believe this CMO has adequate capacity to produce the volumes needed to
meet near-term projected demand for the commercial launch of DefenCath. We have also received FDA approval for finished dosage manufacturing
of DefenCath from Siegfried Hameln.
We
have no direct control over the manufacturing of DefenCath or our product lines. If the contract manufacturers are unable to produce
sufficient quantities of DefenCath or our product lines, as a result of a lack of available materials, supply chain delays or otherwise,
then we would need to identify and contract with additional or replacement third-party manufacturers. Additionally, if the manufacturers
are not able to quickly scale production to algin with rapid changes in demand, our results of operations may be negatively impacted.
If we are unable to identify suitable additional or replacement third-party manufacturers, or are only able to do so on unfavorable terms,
our ability to commercialize DefenCath and our future profitability would be adversely affected.
In addition, we have no direct
control over manufacturing costs of DefenCath or our product lines. If the cost of manufacturing increases, or if the cost of the materials
used increases, these costs will be passed on to us, making the cost of clinical trials and commercializing DefenCath and our product
lines more expensive. Increases in manufacturing costs could adversely affect our future profitability if we are unable to pass all of
the increased costs along to our customers.
Our continuing reliance on
third parties for manufacturing entails a number of additional risks, including reliance on third parties for legal and regulatory compliance
and quality assurance, the possible breach of the manufacturing or supply agreement by such third parties, and the possible termination
or nonrenewal of the agreement by such third parties at a time that is costly or inconvenient for the Company. Further, we, along with
our contract manufacturers, are required to comply with FDA requirements for cGMPs, related to product testing, quality assurance, manufacturing
and documentation. Our contract manufacturers may fail to comply with the applicable FDA regulatory requirements, which could result
in delays to our product development programs, result in adverse regulatory actions against them or us, and prevent us from ultimately
receiving product marketing approval. They also generally must pass an FDA preapproval inspection for conformity with cGMPs before we
can obtain approval to manufacture our product lines and will be subject to ongoing, periodic, unannounced inspection by the FDA and
corresponding state agencies to ensure strict compliance with cGMP and other applicable government regulations and corresponding foreign
standards. Not complying with FDA requirements could result in a product recall or prevent commercialization of our product lines and
delay our business development activities. In addition, such failure could be the basis for the FDA to issue a warning or untitled letter
or take other regulatory or legal enforcement action, including recall or seizure, total or partial suspension of production, suspension
of ongoing clinical trials, refusal to approve pending applications or supplemental applications, and potentially civil and/or criminal
penalties depending on the matter. Similarly, we, along with our contract manufacturers, are required to comply with all applicable healthcare
laws and regulations, such as, without limitation, the federal AKS, the civil False Claims Act, and civil monetary penalty laws, as well
as similar state laws. Violation of any such laws by a contract manufacturer could materially impact our operations.
We rely on third parties to conduct our clinical trials and
pre-clinical studies. If those parties do not successfully carry out their contractual duties or meet expected deadlines, our product
lines may not advance in a timely manner or at all.
In the course of our pre-clinical
and clinical trials, we may rely on third parties, including laboratories, investigators, and manufacturers, to perform critical services
for us, many of which are required to be conducted consistent with regulations on Good Laboratory Practice (“GLP”). Study
sites are responsible for many aspects of the trials, including finding and enrolling subjects for testing and administering the trials.
Although we may rely on these third parties to conduct our pre-clinical and clinical trials, we are responsible for ensuring that each
of our trials is conducted in accordance with its investigational plan and protocol and that the integrity of the studies and resulting
data is protected. Moreover, the FDA and foreign regulatory authorities require us to comply with regulations and standards, commonly
referred to as Good Clinical Practices (“GCPs”), for conducting, monitoring, recording, and reporting the results of clinical
trials to ensure that the data and results are scientifically credible and accurate, and that the trial subjects are adequately informed
of the potential risks of participating in such trials. Our reliance on third parties does not relieve us of these responsibilities and
requirements. These third parties may not be available when we need them or, if they are available, may not comply with all regulatory
and contractual requirements or may not otherwise perform their services in a timely or acceptable manner, and we may need to enter into
new arrangements with alternative third parties and our clinical trials may be extended, delayed or terminated. These independent third
parties may also have relationships with other commercial entities, some of which may compete with us. In addition, if such third parties
fail to perform their obligations in compliance with our protocols or the applicable regulatory requirements, our trials may not meet
regulatory requirements or may need to be repeated, we may not receive marketing approvals, or we or such third parties may face regulatory
enforcement. As a result of our dependence on third parties, we may face delays, failures or cost increases outside of our direct control.
These risks also apply to the development activities of collaborators, and we do not control their research and development, clinical
trial or regulatory activities.
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Risks Related to our Common Stock
Our executive officers and directors may
exercise stock options and sell shares of their stock, and these events could adversely affect our stock price.
Sales of our common stock
and exercise of stock options by our executive officers and directors, or the perception that such events may occur, could adversely
affect the market price of our common stock. Our executive officers and directors may sell stock in the future, either as part, or outside,
of trading plans under Rule 10b5-1 under the Exchange Act.
Our common stock price has fluctuated considerably
and is likely to remain volatile, in part due to the limited market for our common stock and you could lose all or a part of your investment.
From December 31, 2023, through
December 31, 2024, the high and low sales prices for our common stock were $13.85 and $2.89, respectively.
The market price of our common
stock has fluctuated considerably and may continue to fluctuate significantly in response to a number of factors, some of which are beyond
our control.
In addition, the stock markets
in general, and the stock of pharmaceutical and medical device companies in particular, have experienced extreme price and volume fluctuations
that have often been unrelated or disproportionate to the operating performance of these companies. In addition, changes in economic
conditions in the U.S., the European Union or globally, particularly in the context of current global events, could impact upon our ability
to grow profitably. Adverse economic changes are outside our control and may result in material adverse impacts on our business or our
results of operations. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our
actual operating performance. In the past, following periods of volatility in the market price of a company’s securities, securities
class-action litigation has often been instituted against that company. Such litigation, if instituted against us, could cause us to
incur substantial costs and divert management’s attention and resources.
For these reasons and others,
an investment in our securities is risky and you should invest only if you can withstand wide fluctuations in and a significant or complete
loss of the value of your investment.
A significant number of additional shares
of our common stock may be issued at a later date, and their sale could depress the market price of our common stock.
As of December 31, 2024,
we had outstanding the following securities that are convertible into or exercisable for shares of our common stock:
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Additionally, there are 4,756,909
shares of common stock available for grants under the Amended and Restated 2019 Omnibus Stock Plan (adopted on October 13, 2022 and amended
on November 21, 2024).
The possibility of the issuance
of these shares, as well as the actual sale of such shares, could substantially reduce the market price for our common stock and impede
our ability to obtain future financing.
Our internal control over financial reporting
and our disclosure controls and procedures may not prevent all possible errors that could occur.